The FDA would only withdraw a rare disease gene therapy for “pretty clear” reasons, such as when there is minimal benefit with extensive side effects. The one-and-done nature of administration also makes withdrawal easier, CBER Director Peter Marks says.

Gestational age and a binary, neonatal composite endpoint are insufficient to assess outcomes that are important to babies and families, clinicians and researchers say, suggesting development of continuous variables or scales with weighted components and a comparison of outcomes among newborns in gestational age ‘buckets.’

OCE Director Richard Pazdur was less sanguine, however, noting that while the Pepaxto withdrawal may have been faster than others, it still required more than 60 meetings to complete.

In its latest batch of product-specific guidances, the FDA released multiple new and revised guidances on inhalers, including several on GSK’s products such as Relenza and the Ellipta trio.